Disorder of GPI anchor biosynthesis

This completed Phase 3 trial tested ravulizumab in 18 adults in China with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study measured changes in LDH, a marker of red blood cell breakdown, to see if the drug could control the…

This study tested the long-term safety of a drug called pegcetacoplan in people with PNH, a rare blood disease that destroys red blood cells. All 137 participants had already completed a prior pegcetacoplan study and continued treatment. The main goal was to track side effects ov…

This study tested eculizumab in 25 adults with PNH in China who had not previously taken complement inhibitors. Participants received infusions over 62 weeks. The main goal was to see if the drug reduced LDH levels, a marker of disease activity. Results showed a decrease in LDH, …

This early study tested an experimental drug called OMS906 (zaltenibart) in 15 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease where red blood cells break apart. The main goal was to check safety and how the body handles the drug. Researchers also look…

This study followed 120 adults with paroxysmal nocturnal hemoglobinuria (PNH) who switched from the older drug eculizumab to the newer ravulizumab. Researchers measured changes in a blood marker called LDH to see if the new drug controlled red blood cell breakdown just as well or…

This phase 2 trial tested a combination of treosulfan, fludarabine, and rabbit antithymocyte globulin before a bone marrow transplant in 40 people with bone marrow failure diseases like Diamond-Blackfan anemia and Shwachman-Diamond syndrome. The goal was to see if this conditioni…

This study tested a new tablet called HSK39297 in 36 adults with paroxysmal nocturnal hemoglobinuria (PNH) who still had anemia despite stable treatment with anti-C5 antibodies. The goal was to see if the pill could raise hemoglobin levels and reduce the need for blood transfusio…

This study tested an oral medication called iptacopan in 52 adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) who were already stable on standard infusion treatments. Participants switched from their usual therapy to iptacopan pills taken twice daily. The main goal was to see…

This phase 3 trial tested a new drug called HRS-5965 in 39 adults with paroxysmal nocturnal hemoglobinuria (PNH) who remained anemic even after standard anti-C5 antibody treatment. The study aimed to see if the drug could raise hemoglobin levels without needing blood transfusions…

This study tested a new pill called HSK39297 against a standard infusion drug (eculizumab) in 73 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. Participants had not received prior complement inhibitor treatment. The mai…

This study tested a new drug called OMS906 in 12 adults with paroxysmal nocturnal hemoglobinuria (PNH) who were not getting enough benefit from the standard treatment ravulizumab. The goal was to see if OMS906 is safe and can improve hemoglobin levels. Participants received OMS90…

This Phase 3 study tested a new oral medication, HRS-5965, against the standard infusion eculizumab in 76 adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The main goal was to see if HRS-5965 could raise hemoglobin levels to normal without needing blo…

This completed study looked at red and white blood cells in people with a rare blood disorder called PNH who also have bone marrow failure. Over 5,500 participants aged 10 and older with aplastic anemia or related conditions provided blood samples. The goal was simply to learn mo…

This study collected health information from over 6,000 people with Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare blood disorder. The goal was to track the safety of two medications, Soliris and Ultomiris, and to understand how PNH changes over time. Participants were followe…

This completed study followed 724 people in China with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. Researchers aimed to better understand how the disease progresses and how patients respond to different treatments, including the newly available drug eculizum…

This early-stage study tested a new drug, LP-005, in 68 healthy adults aged 18 to 50. The main goal was to check if the drug is safe and how the body handles it. This information will help design future studies for people with a rare blood disorder called PNH.

This study looked at how many girls and young women aged 11 to 20 with chronic diseases (like diabetes or immune conditions) got the HPV vaccine, compared to those without chronic illness. Researchers reviewed records of 223 participants from a hospital in France. The goal was to…